Preclinical evaluation of multiple species of PEGylated recombinant phenylalanine ammonia lyase for the treatment of phenylketonuria

Phenylketonuria (PKU) is a metabolic disorder, in which loss of phenylalanine hydroxylase activity results in neurotoxic levels of phenylalanine. We used the Pah(enu2/enu2) PKU mouse model in short- and long-term studies of enzyme substitution therapy with PEGylated phenylalanine ammonia lyase (PEG-...

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Hlavní autoři: Sarkissian, Christineh N., Gámez, Alejandra, Wang, Lin, Charbonneau, Marilyse, Fitzpatrick, Paul, Lemontt, Jeffrey F., Zhao, Bin, Vellard, Michael, Bell, Sean M., Henschell, Carroll, Lambert, Amy, Tsuruda, Laurie, Stevens, Raymond C., Scriver, Charles R.
Médium: Artigo
Jazyk:Inglês
Vydáno: National Academy of Sciences 2008
Témata:
Biological Sciences
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC2634911/
https://ncbi.nlm.nih.gov/pubmed/19095795
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1073/pnas.0808421105
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